Omaveloxolone for Friedreich's Ataxia
Trial Summary
What is the purpose of this trial?
This trial is testing omaveloxolone, a drug that may help people with Friedreich's ataxia by boosting their body's natural defenses against cell damage. The study focuses on patients with this genetic condition because they have weakened antioxidant systems. Omaveloxolone works by activating a protein that helps protect cells from damage. It aims to improve cell function and reduce harmful effects in people with Friedreich's ataxia.
Do I need to stop my current medications for the trial?
You may need to stop taking certain medications before joining the trial. Specifically, you cannot take certain drugs that affect liver enzymes or are processed by specific pathways in the body within 7 days before starting the study. It's best to discuss your current medications with the study team to see if any need to be stopped.
What data supports the effectiveness of the drug Omaveloxolone for Friedreich's Ataxia?
Omaveloxolone has been shown to activate the Nrf2 pathway, which helps reduce oxidative stress and improve mitochondrial function in Friedreich's Ataxia, a condition with no approved treatments. Clinical trials have demonstrated its potential benefits in improving symptoms by targeting dysfunctional pathways in the disease.12345
Is omaveloxolone safe for humans?
How is the drug omaveloxolone unique in treating Friedreich's ataxia?
Omaveloxolone is unique because it activates the Nrf2 pathway, which helps reduce oxidative stress and improve mitochondrial function, addressing key issues in Friedreich's ataxia. Unlike other treatments, it is an oral medication specifically approved for this condition, offering a novel approach by targeting the underlying cellular dysfunction.12345
Eligibility Criteria
This trial is for people aged 16-40 with genetically confirmed Friedreich's ataxia, who can swallow capsules and have a specific level of disease severity (modified FARS score between 20 and 80). Participants should be stable in their exercise routine and able to complete maximal exercise testing. They cannot join if they've used certain drugs recently, have significant heart disease or abnormal blood tests, uncontrolled diabetes, cognitive impairments, active infections, or substance abuse issues.Inclusion Criteria
Exclusion Criteria
Timeline
Screening
Participants are screened for eligibility to participate in the trial
Treatment Part 1
Randomized, placebo-controlled, double-blind, dose-escalation study to evaluate the safety of omaveloxolone (RTA 408) at various doses
Treatment Part 2
Randomized, placebo-controlled, double-blind, parallel-group study to evaluate the safety and efficacy of omaveloxolone (RTA 408) 150 mg
Extension
Long-term safety and tolerability of omaveloxolone (RTA 408) in qualified participants, open-label
Follow-up
Participants are monitored for safety and effectiveness after treatment
Treatment Details
Interventions
- Omaveloxolone
Omaveloxolone is already approved in United States, European Union for the following indications:
- Friedreich's ataxia
- Friedreich's ataxia
Find a Clinic Near You
Who Is Running the Clinical Trial?
Biogen
Lead Sponsor
Daniel Quirk
Biogen
Chief Medical Officer
MD
Christopher A. Viehbacher
Biogen
Chief Executive Officer since 2022
Graduated from Queen's University, Kingston, Ontario, Canada
Reata, a wholly owned subsidiary of Biogen
Lead Sponsor
Reata Pharmaceuticals, Inc.
Lead Sponsor
Friedreich's Ataxia Research Alliance
Collaborator
AbbVie
Industry Sponsor
Dr. Roopal Thakkar
AbbVie
Chief Medical Officer since 2023
MD from Wayne State University School of Medicine
Robert A. Michael
AbbVie
Chief Executive Officer
Bachelor's degree in Finance from the University of Illinois